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EN7

Development stage: Preclinical
Modality: Small Molecules

Overview

EN7 is a small molecule that functions as a selective inhibitor of Tubulin tyrosine ligase-like protein 4 (Ttll4). Ttll4 catalyzes the post-translational glutamylation of nucleophosmin (NPM1c), a mutated protein found in acute myeloid leukemia (AML) patients. By inhibiting Ttll4, EN7 reduces NPM1c glutamylation, which in turn impairs proliferation and promotes myeloid differentiation in NPM1c-mutated AML cell lines. Importantly, EN7 does not exhibit these effects on wild-type NPM1 cell lines or healthy CD34+ bone marrow progenitors, suggesting a potential therapeutic window for targeting Ttll4 in NPM1c AML.

Targets

TTLL4

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